CONVENTIONAL APPROACHES TO THE THERAPY OF HEREDITARY MYOPATHIES

The aim of the work was to analyze available therapeutic options for conventional therapy hereditary myopathies. Materials and methods. When searching material writing a review article, such abstract databases as PubMed Google Scholar were used. search carried out on publications during period from 1980 September 2022. following words their combinations selected parameters literature selection: “myopathy”, “Duchenne”, “myodystrophy”, “metabolic”, “mitochondrial”, “congenital”, “symptoms”, “replacement”, “recombinant”, “corticosteroids”, “vitamins”, “tirasemtiv”, “therapy”, “treatment”, “evidence”, “clinical trials”, “patients”, “dichloracetate”. Results. Congenital myopathies are heterogeneous group pathologies that caused by atrophy degeneration muscle fibers due mutations in genes. Based number clinical pathogenetic features, divided into: 1) congenital myopathies; 2) muscular dystrophy; 3) mitochondrial 4) metabolic At same time, treatment approaches vary significantly depending type myopathy can be based substitution mutant protein; an increase its expression; stimulation internal compensatory pathways restoration compounds balance associated with protein function (for enzymes); 5) impact (with myopathies); 6) reduction inflammation fibrosis dystrophies); well 7) mass strength. current presents data each listed approaches, specific pharmacological agents description action mechanisms. Conclusion. Currently, groups used or undergoing trials various types: inotropic, anti-inflammatory antifibrotic drugs, antimyostatin drugs promote translation through stop codons (applicable nonsense mutations). In addition, enzyme cofactors, biogenesis stimulators, antioxidants treat Finally, recombinant alglucosidase avalglucosidase have been clinically approved replacement (Pompe’s disease).